At the 5th annual Patient-Centered Oncology Care® meeting, hosted by The American Journal of Managed Care®, experts discussed the contradiction presented by immuno-oncology agents in the world of precision medicine.

Paying for CGP and Immunotherapy

When asked about payer response to these new genomic technologies, Fabrizio said that payers have been slow to adopt these advancements, but that they are being more proactive and more engaging in discussions with their company. “I see this as an encouraging sign for the most efficient healthcare possible,” he added. Porter also emphasized that because of the scale at which the chimeric antigen receptor (CAR) treatment is being developed, it ends up being very expensive. The treatment is being personalized for each individual patient, with strong biotechnology input. “Third-party payers have not at all been involved in paying for this, although they have been asked to pay for the standard-of-care treatment,” he added. “Supportive care is not really being denied by payers, but academic institutions cannot fund this type of care…the biggest grants can maybe cover a couple patients a year.”

Porter strongly believes that the pharmaceutical and biotechnology industry would have to provide maximal support for more clinical use of this technology. “We are breaking new ground here and we’d like to see more collaboration,” he added. As of now, CAR treatment remains in the trial stage.

Can the FDA lend any support with helping payers navigate these novel technologies to develop payment policies?

“The FDA does not traditionally involve itself in cost mandates, but we are thinking about value across the entire spectrum of drug development,” Khozin said, adding that Richard Pazdur, MD, who heads the OCE, is thinking of collaborating with the National Cancer Institute and CMS on value creation, not the cost of drugs. When asked to provide a view of the direction toward which the field was moving, Fabrizio said that a more personalized approach for immunotherapy was necessary, such as using CGP to uncover neoantigenic epitopes. He added, however, that deciding on trial endpoints is a challenge, especially with a smaller batch of trial participants, the direction toward which the field seems to be moving. “From a diagnostic test point of view, we want to understand actionable genomic targets, such as tumor mutation burden or identifying neoantigens.”

According to Porter, CAR-T is “a once-and-done therapy.”This raises the potential for alternate trial endpoints, such as response rate and achieving minimal residual disease status.

Khozin clarified that the FDA does not always push for overall survival. “We sometimes hear that sponsors say they have to do randomized studies for payers or other agencies…but we ask them not to conduct randomized clinical trials.” Referencing the cobas liquid biopsy companion diagnostic that was approved last year,2 Khozin said that the FDA has received several other proposals for such liquid biopsy tests, which point to using the levels of circulating tumor cells as a surrogate endpoint.

Porter summed the discussion by saying, “This era of immuno-oncology is revolutionary. It is one of the most exciting times in oncology, and the rate of change is staggering—from a clinical trial, regulatory, and data standpoint. Information is being generated at a very fast pace and needs rapid dissemination, and we need continuous conversation among the various entities to keep the progress going.”

REFERENCES

Dangi-Garimella, S. 5 Ongoing Initiatives at the FDA. The American Journal of Managed Care® website. http://www.ajmc.com/newsroom/5-initiatives-catching-speed-at-the-fda. Published August 26, 2016. Accessed November 18, 2016.

Dangi-Garimella, S. First Liquid Biopsy Companion Diagnostic, for Erlotinib, Approved by FDA. The American Journal of Managed Care® website. http://www.ajmc.com/newsroom/first-liquid-biopsy-companion-diagnostic-for-erlotinib-approved-by-fda. Published June 2, 2016. Accessed November 18, 2016.